AU2012229330B2 - Dosing regimens for the treatment of Fabry disease - Google Patents

Dosing regimens for the treatment of Fabry disease Download PDF

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Publication number
AU2012229330B2
AU2012229330B2 AU2012229330A AU2012229330A AU2012229330B2 AU 2012229330 B2 AU2012229330 B2 AU 2012229330B2 AU 2012229330 A AU2012229330 A AU 2012229330A AU 2012229330 A AU2012229330 A AU 2012229330A AU 2012229330 B2 AU2012229330 B2 AU 2012229330B2
Authority
AU
Australia
Prior art keywords
gal
agalsidase
migalastat
deoxygalactonojirimycin
administration
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
AU2012229330A
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English (en)
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AU2012229330A1 (en
Inventor
Douglas Stuart Greene
Kenneth Joseph Valenzano
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Amicus Therapeutics Inc
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46831259&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AU2012229330(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of AU2012229330A1 publication Critical patent/AU2012229330A1/en
Application granted granted Critical
Publication of AU2012229330B2 publication Critical patent/AU2012229330B2/en
Priority to AU2016204445A priority Critical patent/AU2016204445B2/en
Priority to AU2018201637A priority patent/AU2018201637A1/en
Priority to AU2020201715A priority patent/AU2020201715A1/en
Priority to AU2022203011A priority patent/AU2022203011A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01062—Glycosylceramidase (3.2.1.62)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Cardiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Hydrogenated Pyridines (AREA)
AU2012229330A 2011-03-11 2012-03-08 Dosing regimens for the treatment of Fabry disease Active AU2012229330B2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2016204445A AU2016204445B2 (en) 2011-03-11 2016-06-28 Dosing regimens for the treatment of fabry disease
AU2018201637A AU2018201637A1 (en) 2011-03-11 2018-03-07 Dosing regimens for the treatment of fabry disease
AU2020201715A AU2020201715A1 (en) 2011-03-11 2020-03-09 Dosing regimens for the treatment of fabry disease
AU2022203011A AU2022203011A1 (en) 2011-03-11 2022-05-05 Dosing regimens for the treatment of fabry disease

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161451798P 2011-03-11 2011-03-11
US61/451,798 2011-03-11
US201161578201P 2011-12-20 2011-12-20
US61/578,201 2011-12-20
US201261596165P 2012-02-07 2012-02-07
US61/596,165 2012-02-07
PCT/US2012/028260 WO2012125402A2 (en) 2011-03-11 2012-03-08 Dosing regimens for the treatment of fabry disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2016204445A Division AU2016204445B2 (en) 2011-03-11 2016-06-28 Dosing regimens for the treatment of fabry disease

Publications (2)

Publication Number Publication Date
AU2012229330A1 AU2012229330A1 (en) 2013-10-03
AU2012229330B2 true AU2012229330B2 (en) 2016-04-21

Family

ID=46831259

Family Applications (5)

Application Number Title Priority Date Filing Date
AU2012229330A Active AU2012229330B2 (en) 2011-03-11 2012-03-08 Dosing regimens for the treatment of Fabry disease
AU2016204445A Active AU2016204445B2 (en) 2011-03-11 2016-06-28 Dosing regimens for the treatment of fabry disease
AU2018201637A Abandoned AU2018201637A1 (en) 2011-03-11 2018-03-07 Dosing regimens for the treatment of fabry disease
AU2020201715A Abandoned AU2020201715A1 (en) 2011-03-11 2020-03-09 Dosing regimens for the treatment of fabry disease
AU2022203011A Pending AU2022203011A1 (en) 2011-03-11 2022-05-05 Dosing regimens for the treatment of fabry disease

Family Applications After (4)

Application Number Title Priority Date Filing Date
AU2016204445A Active AU2016204445B2 (en) 2011-03-11 2016-06-28 Dosing regimens for the treatment of fabry disease
AU2018201637A Abandoned AU2018201637A1 (en) 2011-03-11 2018-03-07 Dosing regimens for the treatment of fabry disease
AU2020201715A Abandoned AU2020201715A1 (en) 2011-03-11 2020-03-09 Dosing regimens for the treatment of fabry disease
AU2022203011A Pending AU2022203011A1 (en) 2011-03-11 2022-05-05 Dosing regimens for the treatment of fabry disease

Country Status (16)

Country Link
US (4) US20140219986A1 (zh)
EP (2) EP2683382B1 (zh)
JP (5) JP2014528901A (zh)
KR (1) KR20140011367A (zh)
CN (2) CN107088225A (zh)
AU (5) AU2012229330B2 (zh)
CA (1) CA2829947C (zh)
CL (1) CL2013002601A1 (zh)
EA (1) EA031874B1 (zh)
ES (1) ES2807502T3 (zh)
HK (1) HK1242998A1 (zh)
MX (1) MX2013010446A (zh)
SG (2) SG193379A1 (zh)
TW (3) TW201740945A (zh)
WO (1) WO2012125402A2 (zh)
ZA (1) ZA201306735B (zh)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2533050T6 (da) 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
HUE051377T2 (hu) 2008-02-12 2021-03-01 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
UY34317A (es) 2011-09-12 2013-02-28 Genzyme Corp Anticuerpo antireceptor de célula T (alfa)/ß
BR112015020885A2 (pt) 2013-03-11 2017-10-10 Genzyme Corp polipeptídeos de ligação hiperglicosilados
AU2015216909B2 (en) * 2014-02-17 2017-07-20 Glaxosmithkline Intellectual Property Development Limited Microbiological process
EP3129067B1 (en) 2014-03-19 2023-01-04 Genzyme Corporation Site-specific glycoengineering of targeting moieties
US9675627B2 (en) * 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7001593B2 (ja) 2015-08-11 2022-01-19 コグノア， インコーポレイテッド 人工知能およびユーザ入力を用いて発達進度を判定するための方法および装置
US11972336B2 (en) 2015-12-18 2024-04-30 Cognoa, Inc. Machine learning platform and system for data analysis
EP3432882B1 (en) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
US20190183869A1 (en) * 2016-07-19 2019-06-20 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients
CA3053245A1 (en) 2017-02-09 2018-08-16 Cognoa, Inc. Platform and system for digital personalized medicine
EP4324522A2 (en) * 2017-05-30 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
KR102631805B1 (ko) 2017-05-30 2024-01-31 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP7066157B2 (ja) 2017-06-14 2022-05-13 学校法人 明治薬科大学 ファブリー病治療用医薬の組合せ物及びその利用
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
US20210315875A1 (en) * 2018-08-20 2021-10-14 Amicus Therapeutics, Inc. Methods of Treating Fabry Disease in Patients Having a Mutation in the GLA Gene
JP2022527946A (ja) 2019-03-22 2022-06-07 コグノア，インク． 個別化されたデジタル治療方法およびデバイス
CA3141226A1 (en) * 2019-06-11 2020-12-17 Franklin Johnson Methods of treating fabry disease in patients having renal impairment
US11833164B2 (en) 2019-08-07 2023-12-05 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999062517A1 (en) * 1998-06-01 1999-12-09 Fan Jian Qiang METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A
US20060153829A1 (en) * 2003-01-31 2006-07-13 Mount Sinai School Of Medicine Of New York University Stable formulations of purified proteins
WO2008121826A2 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
US20100266571A1 (en) * 2007-04-26 2010-10-21 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100457106C (zh) * 2005-06-15 2009-02-04 同济大学 1-脱氧野尻霉素在制备治疗糖尿病肾病药物中的应用
DK2533050T6 (da) * 2006-05-16 2015-09-21 Amicus Therapeutics Inc Behandlingsmuligheder ved Fabrys sygdom
WO2009032171A1 (en) * 2007-08-29 2009-03-12 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999062517A1 (en) * 1998-06-01 1999-12-09 Fan Jian Qiang METHOD OF ENHANCING LYSOSOMAL α-GALACTOSIDASE A
US20060153829A1 (en) * 2003-01-31 2006-07-13 Mount Sinai School Of Medicine Of New York University Stable formulations of purified proteins
WO2008121826A2 (en) * 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
US20100266571A1 (en) * 2007-04-26 2010-10-21 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

Also Published As

Publication number Publication date
EP2683382B1 (en) 2020-04-22
AU2020201715A1 (en) 2020-04-09
TW201302197A (zh) 2013-01-16
TW201634048A (zh) 2016-10-01
EP2683382A4 (en) 2014-10-22
CA2829947C (en) 2019-01-15
AU2016204445A1 (en) 2016-07-21
AU2022203011A1 (en) 2022-05-26
US20200268890A1 (en) 2020-08-27
CN103974619B (zh) 2017-02-15
EA201301018A1 (ru) 2014-04-30
EP3698792A1 (en) 2020-08-26
ZA201306735B (en) 2014-07-30
TWI624258B (zh) 2018-05-21
CL2013002601A1 (es) 2014-03-28
JP2021098697A (ja) 2021-07-01
SG193379A1 (en) 2013-10-30
TW201740945A (zh) 2017-12-01
JP2017132780A (ja) 2017-08-03
NZ615726A (en) 2015-01-30
US20140219986A1 (en) 2014-08-07
AU2016204445B2 (en) 2018-03-22
EP2683382A2 (en) 2014-01-15
EA031874B1 (ru) 2019-03-29
WO2012125402A2 (en) 2012-09-20
WO2012125402A3 (en) 2014-05-01
AU2018201637A1 (en) 2018-04-05
JP2023093448A (ja) 2023-07-04
JP2020033360A (ja) 2020-03-05
TWI624259B (zh) 2018-05-21
SG10201604757RA (en) 2016-08-30
CN103974619A (zh) 2014-08-06
HK1242998A1 (zh) 2018-07-06
JP2014528901A (ja) 2014-10-30
US20240115708A1 (en) 2024-04-11
ES2807502T3 (es) 2021-02-23
MX2013010446A (es) 2014-03-27
AU2012229330A1 (en) 2013-10-03
KR20140011367A (ko) 2014-01-28
CN107088225A (zh) 2017-08-25
US20180153999A1 (en) 2018-06-07
CA2829947A1 (en) 2012-09-20

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